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Roll up your sleeves for a two-day, introductory workshop on how to prepare an effective electronic Common Technical Document (eCTD) submission. This in-person workshop includes skill-building, interactive exercises, case studies and small group discussions.

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Take 15 minutes to learn how to apply regulatory competency frameworks to your professional development plan with a free webinar from RAPS. From entry level to C-suite executives, RAPS understands the complexity of the regulatory profession and has a free tool to help you build out a roadmap for career success.

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Featured Article

An
overview of expedited pathways for US medical devices that are alternatives to Traditional
510(k)s and Premarket Approval Applications (PMAs). These include Special
510(k), Abbreviated 510(k) and Humanitarian Device Exemption (HDE) submission types.

Since early 2014, more than 20 states have introduced so-called "Right to Try" bills in the hopes of allowing terminally ill patients to access experimental—and potentially life-saving—treatments more easily. These bills are modeled off a federal policy known as "Compassionate Use," but contain several key changes meant to make it faster and easier for patients to obtain experimental therapies.

FDA Calls for Limited Clinical Trials Transparency through Use of De-Identified and Masked Data

The US Food and Drug Administration (FDA) is asking for input from industry and the public regarding a proposal to make de-identified and masked data taken from medical product applications available to select members of the public, a massive change in policy it says is driven by its desire to improve the product development process.

Background

The proposal, announced on 3 June 2013, follows a lengthy push by FDA Commissioner Margaret Hamburg to improve how FDA leverages regulatory science to the benefit of product applications. That push, known as the Regulatory Science Initiative, is specifically meant to "enhance the science and knowledge critical to improving the development, manufacture, evaluation, and safe use of critically needed new therapies," FDA explained in the announcement.

And that's where preclinical and clinical study data comes into play, FDA continued. The agency explained that data from one study may be useful to addressing "key hurdles in drug development" such as the development of new valid endpoints for trials, the predictive value of preclinical models, a deeper understanding of how products affect different diseases, and whether new clinical designs could be used.

Regulators provided the example of a hepatitis C treatment, which had previously relied upon disease detection at 24 weeks. An analysis of data across 15 trials allowed FDA to determine that patient response at 12 weeks was just as indicative of the ultimate response, allowing companies to cut the trial time in half.

Safety issues, too, can often be addressed more easily when data is pooled. FDA said an analysis of 199 trials of 11 antiepileptic drugs identified an increased risk of suicidal ideation and behavior for patients. Other trials have provided key data on which biomarkers predict hepatic toxicity.

All of this serves to benefit both patients and companies alike, FDA said. Patients would benefit from having earlier access to safer medicines, while companies would be able to save money and get an earlier return on their investment.

Difficulties

There's just one problem, says FDA: While it has considerable expertise in analyzing this type of data, it says it wants to bring in additional, outside review resources in the form of independent experts.

As with other clinical trials transparency efforts, the toughest question remains how FDA can bring in outside experts without violating the privacy rights of patients involved in the studies while at the same time protecting the commercial investments of sponsors.

Despite those difficult issues, FDA says it intends to move forward with its proposal with an open ear toward the concerns of sponsors. "FDA intends to consider the extent and nature of public availability of de-identified and masked subject level data necessary to achieve specific aims," it wrote in its Federal Register posting-data that is neither linkable to a specific product nor a specific application nor individual patients.

How the agency will mask data is not yet certain, but it said it is considering a range of strategies, including making limited data available to researchers via a random sample or a subset, restricting data analysis to limited data fields, and pooling data to obscure the products.

FDA's proposal is similar in many respects to an announcement made in October 2012 by GlaxoSmithKline, which mentioned that it would allow limited researchers to become involved with analyzing its data, and that it, too, would be working to anonymize patient-level data.

FDA said in its statement that it has an "unwavering commitment" to patient privacy and "will not make available business-related confidential commercial information contained in product applications" such as licensing agreements or information identifying suppliers.

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